Achieving regulatory success in cell therapy

写的:

Cell therapies are transforming the treatment of cancer; however, 它们的革命性地位给现有的主要用于评估合成分子和已建立的生物制剂(如治疗性抗体)的监管框架带来了独特的挑战. 澳门葡京赌博游戏致力于细胞治疗的未来——这意味着与监管机构合作，开拓设计这些“活药物”的新技术。, 把它们带给更多的病人.

Cell therapies present unique regulatory challenges

作为“活的药物”, 细胞疗法需要一个独立的监管框架，近年来一直在发展.

生产需求 因为细胞疗法很复杂, 需要专门的洁净室和符合GMP的设施, stringent quality control and additional regulatory considerations.

自体细胞治疗产品是由患者自身的免疫细胞产生的，这是固有的 更多的变量 than other products synthesised at large scale in a laboratory, 比如小分子和抗体. 因此, key steps in cell therapy development—from collecting a patient’s cells, through to engineering and manufacturing a product—are even more important to regulators.

Additionally, cell therapies are often administered as a single, long-lived infusion. This means regulators are extremely interested in close monitoring of 长期影响，因为它们很难预测.¹ 结果是, 与其他药物相比，细胞疗法对患者长期随访的要求更为严格——强制随访期为15年.² This can have important implications for long-term patient care.

Working with regulators to evolve research regulations

We see the conversation between regulators and industry as an evidence-led, 合作过程. 双方的共同目标是改变监管环境，从而促进可能改变患者生活的药物的开发.

We are working with regulators to simplify and 简化 细胞治疗制造. 这包括实施旨在缩短过程并保持自体细胞疗法“类似干细胞”的新技术。, with the aim of optimising the safety profile of cell therapies.

从健康供体中提取的同种异体或“现成”细胞疗法的出现可能会带来这一结果 减轻细胞变异性, and conversations are ongoing around rejection prevention, 专注于组织相容性，并从移植和其他治疗领域学习.

最后, 澳门葡京赌博游戏正在研究分子“控制开关”，以便在给药后打开和关闭细胞疗法——如果需要的话——目标是 降低长期安全风险 细胞疗法.

Regulators are taking the lead in outlining a pragmatic and patient benefit-focused approach 对于这些策略, 在第一代获批用于临床的细胞疗法取得成功的基础上, 并帮助开辟一条道路，将这些潜在的变革性药物带给更多的癌症患者.

Global cell therapy regulation standards are converging

有一个 全面的监管框架 对于“高级疗法”，比如细胞疗法，尽管具体情况因地理位置而异.^3,4 有一个n increasing need to develop consistent, 精准及时的监管指导, addressing the nuances unique to cell therapy. Efforts are in motion to align standards worldwide, 目的是简化细胞治疗的发展和改善这些药物的可及性.

的 国际协调理事会 人用药品技术要求(我)已经成立了一个细胞治疗小组，为细胞和基因治疗相关问题的技术讨论提供了一个平台.⁵ This group has now been joined by Chinese regulators, 大大扩大了覆盖面积, to help build a truly global regulatory paradigm.⁶

利益相关者的目标是 统一和精简 全球监管框架，包括来自不同国家的创新和专业知识. 简化的监管途径将确保开发团队能够缩短新药到达患者手中所需的时间.

与监管机构合作 卫生当局简化监管途径对于成功开发和部署细胞疗法来对抗癌症至关重要. 在澳门葡京网赌游戏, 澳门葡京赌博游戏的全球影响力以及对全球和国家一级卫生服务政策的深入了解意味着澳门葡京赌博游戏处于与监管机构在新框架内合作的有利地位.

Future direction for cell therapy regulation

Highlighting our joint aspiration of improving access to new, 革命性的治疗, and based on close collaboration with industry, regulators are facilitating the development of a framework, 支持快速发展的细胞治疗领域的新技术和发展途径.

有一个 positive recent trend towards more collaborative, partnership-based and open-source research and development (R&D), which promises to accelerate and diversify the industry. 澳门葡京赌博游戏是这类开源创新的全球领导者，拥有400多个研发项目的经验&D collaborations, including in cell therapy.

的 wider shift to greater collaboration, while it has the potential to accelerate scientific innovation, 可能会带来监管方面的挑战. 澳门葡京赌博游戏广泛而多样的经验意味着澳门葡京赌博游戏处于帮助发展与协作R相关的监管政策的理想位置&D.⁷

通过数字化革命，R&D正在利用虚拟实验和模拟、数字协作和大数据推进.⁸  ‘Digital’ has the potential to change and accelerate all regulatory processes, including the traditional ways of developing and submitting data to regulatory agencies. 例如:

• 通过“实时肿瘤学评论”⁹ 食品及药物管理局 reviewers are gaining earlier access to data, to identify potential data-quality and review issues, aiming to provide early feedback to allow for a more efficient review process.
• 奥比斯项目,¹⁰ 食品及药物管理局发起的试点项目, 为国际合作伙伴同时提交和审查肿瘤产品提供了一个框架, allowing earlier registrations in countries outside the US.
• We are partnering with organisations such as Accumulus协同作用¹¹ to create digital regulatory submissions while adhering to, 考虑到, the different processes of regulators across the world.

澳门葡京赌博游戏的肿瘤 细胞治疗组 was formed to transform the treatment of hard-to-treat solid tumours, 澳门葡京赌博游戏收购了开拓性的t细胞受体工程公司Neogene，加速了澳门葡京赌博游戏在这一领域的研究. 合作 with regulators to facilitate effective R&D, employ cutting-edge engineering and manufacturing, and develop solution-focused approval processes is a vital part of the solution. We are deeply proud of our progress so far, 很高兴能继续合作，为患者带来最好的治疗方法.

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